1.96E+07 3.9E+07 6.6 E+07 3.4E+07 6.7E+07 5.7E+07 0E+0 1E+7 2E+7 3E+7 4E+7 5E+7 6E07 7E+7 8E+7 9E+7 hWASP1 Cell Growth 234234 Cell Number Days of Growth.

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1.96E E E E E E+07 0E+0 1E+7 2E+7 3E+7 4E+7 5E+7 6E07 7E+7 8E+7 9E+7 hWASP1 Cell Growth Cell Number Days of Growth (post seed) = 4 x10 6 seed, 40 ml, T175 = 2 x10 6 seed, 40 ml, T175 = 8 x10 6 seed, 40 ml, T175 Supplemental Figure 1

1.3E E E E E e+3 1e+4 1e+5 1e+6 1e+7 1e Stability of 650MNDhWASp Titers IU/ml Weeks in culture before 4 day induction Supplemental Figure 2

%Hu CD45 + Cells engrafted in NSG BM 50 a hit MOI 100 1hit + DMSO 1hit + DMSO+ Ex527 1hit + DMSO+ CHIR hit + NAM 2hits MOI 100 1hit MOI hit MOI NAM 1hit MOI 200+ NAM+ SDF1  = 12 methocult cultures %hW + CFU-Cs from NSG BM b. Supplemental Figure 3 p = p = p = p = p = 0.38

Mock +DMSO 650+ DMSO 650+ SDF1  +DMSO nM Rapa for entire culture a. b. Supplemental Figure 4 p = p = p = %CD45 + Cells % Engrafted %hWASP Vector + %CFU-Cs